A CMT Research Foundation–funded project at the University of Illinois Chicago has identified optimal dosing for a potential therapeutic for CMT1X.

Like other forms of CMT, cell-to-cell communication within the peripheral nervous system malfunctions in people with CMT1X, leading to a breakdown in nerve signals resulting in an increase in inflammation, progressive weakness, and loss of sensation in the limbs.  While investigating the role that inflammation may play in the development of CMT symptoms, researchers noted that mice with CMT1X have lower levels of a small anti-inflammatory molecule called inosine. 

Dr. Charles Abrams is currently examining the potential therapeutic use of this molecule in a mouse model of CMT1X. In the first phase of the project, his team tested multiple different doses of inosine and measured how much was able to enter the peripheral nervous system. They have identified the ideal therapeutic dosages and treatment schedule and are now prepared to move forward with the next stage of the project.

In the next phase, Dr. Abrams will evaluate the effects of inosine treatment on the levels of inflammation in the peripheral nervous system of mice with CMT1X. The final phase will involve testing for improvements in symptoms or disease progression. If this work in mice is successful, this project could form the basis for a clinical trial utilizing inosine to treat people with CMT1X.

While inosine is widely available as a supplement and is also being studied as a potential therapeutic for Parkinson’s disease and Multiple Sclerosis, the CMT Research Foundation cautions those with CMT about using inosine before more research is completed.

Project Announcement: https://cmtrf.org/cmt-research-foundation-targets-cmt1x-in-partnership-with-university-of-illinois-chicago/